Fexinidazole

DNDi Achievements

Fexinidazole to treat sleeping sickness Sleeping sickness

First new chemical entity developed by DNDi and the first all-oral cure for all stages of sleeping sickness

2018

Easy-to-use medicine brings treatment closer to patients

NECT, a combination of nifurtimox and eflornithine, was approved for sleeping sickness in 2009, replacing very toxic or complicated drugs. But treatment remained cumbersome, difficult to ship, store, and administer; patients needed to be hospitalized to receive the intravenous infusions and undergo a lumbar puncture to determine the stage of the disease since NECT is only indicated for stage-2.

DNDi, in collaboration with the Swiss Tropical and Public Health, rediscovered the 5-nitroimidazole derivative fexinidazole during a search for compounds with anti-parasitic activity in 2005. The compound had been developed but abandoned for strategic reasons by Hoechst (now Sanofi) in the 1980s. In 2009, DNDi and Sanofi concluded a collaboration agreement.

More than two million people were screened for sleeping sickness as part of three fexinidazole clinical trials, which enrolled 749 patients in the Democratic Republic of Congo and Central African Republic and had to overcome unique challenges of conducting trials in remote areas where sleeping sickness occurs. In the trials, fexinidazole showed high efficacy and safety in both stages of the disease, both in adults and children.

Fexinidazole is the first all-oral treatment for both stages of T. b. gambiense sleeping sickness, the most common form of the disease. Taken as simple pills for 10 days, fexinidazole presents significant advantages over NECT because it eliminates the need for systematic hospitalization and potentially leads to a reduction in the number of lumbar punctures. The European Medicines Agency adopted a positive scientific opinion of fexinidazole in late 2018, paving the way for registration and distribution in endemic countries.

Fexinidazole is the first new chemical entity to have been developed by DNDi, which has steered its progression through all stages of the drug development pipeline from lab to patient.

At a glance

Dosage:

Adults: 3 tablets once-a-day for 4 days, and 2 tablets once-a-day for next 6 days

Children (≥ 6 years old & ≥ 20 kg): 2 tablets once-a-day for 4 days, and 1 tablet once-a-day for next 6 days

Project start: 2005

Impact

Recommended in November 2018 by the European Medicines Agency under Article 58, an innovative regulatory mechanism for the review of new medicines destined for use outside of the European Union

Registration submission in endemic countries underway

Sanofi has committed to donate fexinidazole to WHO, which will distribute it to National Control Programmes in endemic countries

Developed in partnership between DNDi, Sanofi, the HAT Platform, national control programmes of the Democratic Republic of the Congo and Central African Republic, Médecins Sans Frontières, Swiss Tropical & Public Health Institute, with support of the World Health Organization, Department of Control of Neglected Tropical Diseases

“Those affected by sleeping sickness are some of the most vulnerable and live in some of the most remote areas of the Congo, if not the world. They need a treatment that is safe, effective and simple. An all-oral treatment has been a dream of mine for decades. Fexinidazole is a huge leap in how we can tackle this deadly disease.”

Dr Victor Kande, NTD Expert Advisor to the Ministry of Health of DRC and principal investigator of the fexinidazole clinical trials

Funding

Bill & Melinda Gates Foundation, USA; UK aid, UK; Dutch Ministry of Foreign Affairs (DGIS), The Netherlands; Federal Ministry of Education and Research (BMBF) through KfW, Germany; French Development Agency (AFD), France; German Corporation for International Cooperation (GIZ) on behalf of the Government of the Federal Republic of Germany, Germany; Ministry of European and Foreign Affairs (MEAE), France, Médecins sans Frontières; Norwegian Agency for Development Cooperation (Norad), Norway; Republic and Canton of Geneva, Internal Solidarity Office, Switzerland; Spanish Agency for International Development and Cooperation (AECID), Spain; Swiss Agency for Development and Cooperation (SDC), Switzerland; UBS Optimus Foundation, Switzerland; Brian Mercer Charitable Trust, UK; Stavros Niarchos Foundation, USA and other private foundations and individuals from the HAT campaign.

Photo credit: Xavier Vahed-DNDi

Last updated: December 2018

About DNDi

Drugs for Neglected Diseases initiative (DNDi) is a collaborative, patients’ needs-driven, non-profit drug research and development (R&D) organization that is developing new treatments for neglected patients.

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